The Right to Try legislation allows individuals to access investigational therapies outside of clinical trials without approval and consultation from the Food and Drug Administration (FDA).
The law applies to people who have been diagnosed with a life-threatening disease, exhausted approved treatment options, and are unable to participate in a clinical trial involving the investigational drug, as certified by a physician.
The Cystic Fibrosis Foundation is disappointed by the passage of this legislation as it creates additional and undue risks for those seeking access to investigational therapies without addressing the primary access barriers to investigational therapies. Legitimate barriers to such access include a limited supply of the investigational therapy or other concerns from the manufacturer.
Specifically, the Right to Try law:
- Allows unapproved therapies to be given to patients without FDA notification for up to one full year
- Removes the FDA's consultation on dosing, route of administration, dosing schedule, and other important safety measures
- Does not establish any standards for informed consent for patients accessing these therapies
- Prohibits the FDA from halting access to an experimental therapy short of placing a clinical hold on all clinical research related to the therapy
We recognize and share the sense of urgency around developing new and effective treatments for cystic fibrosis and getting them into the hands of people with CF as quickly as possible. Unfortunately, the Right to Try law will not provide greater access to unapproved therapies in the CF drug development pipeline.
The FDA's current expanded access program, though imperfect, facilitates access to investigational therapies outside of clinical trials. Last year, the FDA approved more than 1,800 applications for people facing serious and life-threatening conditions. This is an important way to provide therapies not yet approved by the FDA to those with limited treatment options. The current process requires approval from a physician, the manufacturer, and the FDA.
The FDA approves more than 99 percent of expanded access requests and sometimes makes important dosing and safety improvements to the proposals, which the Right to Try law does not allow. The CF Foundation supports continued use of the FDA's existing expanded access process for people seeking to access investigational therapies.